NCT04295785

Brief Summary

Autoimmune necrotizing myopathies (AINM) in adult patients are characterized by severity of muscle damage, presence of necrosis with little inflammation on muscle biopsy and anti-HMGCR or anti-SRP auto-antibodies. Data on AINM in children are currently lacking. The purpose of this study is to specify the characteristics at AINM diagnosis, treatments and evolution of juvenile AINM with anti-HMGCR or anti-SRP antibodies.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Mar 2020

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 9, 2020

Completed
2 months until next milestone

Study Start

First participant enrolled

March 1, 2020

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 5, 2020

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2020

Completed
Last Updated

March 5, 2020

Status Verified

January 1, 2020

Enrollment Period

8 months

First QC Date

January 9, 2020

Last Update Submit

March 2, 2020

Conditions

Immune-Mediated Necrotizing Myopathy

Keywords

Autoimmune necrotizing myopathyanti-HMGCR antibodiesanti-SRP antibodies

Outcome Measures

Primary Outcomes (8)

  • Type of muscle involvment

    Proximal or distal or axial muscle involvement by muscle testing

    At diagnosis

  • Type of cutaneous involvment

    Rash or Gottron lesions

    At diagnosis

  • Other clinical manifestations

    Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment

    At diagnosis

  • Blood CK level

    Blood CK level

    At diagnosis

  • Histology

    Results of muscle biopsy : necrosis

    At diagnosis

  • Histology

    Results of muscle biopsy : inflammation

    At diagnosis

  • MMT score

    MMT score

    At diagnosis

  • CMAS score

    CMAS score

    At diagnosis

Secondary Outcomes (11)

  • Type of drugs used

    At last visit

  • Iatrogenic complications

    At last visit

  • Iatrogenic complications

    At last visit

  • Status of the disease at last visit

    At last visit

  • Number of relapses

    At last visit

  • +6 more secondary outcomes

Study Arms (1)

autoimmune necrotizing myopathy beginning before 18

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

AINM histologically proved (i.e. predominant muscle fibers necrosis lesions associated with minimal or absent inflammatory lesions), either anti-SRP or anti-HMGCR antibodies positivity and a disease onset before age 18

You may qualify if:

  • AINM histologically proved by predominant muscle fibers necrosis lesions associated with minimal or absent inflammatory lesions
  • Either anti-SRP or anti-HMGCR antibodies positivity
  • Disease onset before age 18

You may not qualify if:

  • Seronegative necrotizing myopathies
  • Forms compatible with another type of myositis (e.g. dermatomyositis)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hopital d'enfants CHRU Nancy

Vandœuvre-lès-Nancy, 54 500, France

Location

Related Publications (8)

  • Benveniste O, Stenzel W, Allenbach Y. Advances in serological diagnostics of inflammatory myopathies. Curr Opin Neurol. 2016 Oct;29(5):662-73. doi: 10.1097/WCO.0000000000000376.

    PMID: 27538058BACKGROUND

  • Mariampillai K, Granger B, Amelin D, Guiguet M, Hachulla E, Maurier F, Meyer A, Tohme A, Charuel JL, Musset L, Allenbach Y, Benveniste O. Development of a New Classification System for Idiopathic Inflammatory Myopathies Based on Clinical Manifestations and Myositis-Specific Autoantibodies. JAMA Neurol. 2018 Dec 1;75(12):1528-1537. doi: 10.1001/jamaneurol.2018.2598.

    PMID: 30208379BACKGROUND

  • Watanabe Y, Uruha A, Suzuki S, Nakahara J, Hamanaka K, Takayama K, Suzuki N, Nishino I. Clinical features and prognosis in anti-SRP and anti-HMGCR necrotising myopathy. J Neurol Neurosurg Psychiatry. 2016 Oct;87(10):1038-44. doi: 10.1136/jnnp-2016-313166. Epub 2016 May 4.

    PMID: 27147697BACKGROUND

  • Allenbach Y, Drouot L, Rigolet A, Charuel JL, Jouen F, Romero NB, Maisonobe T, Dubourg O, Behin A, Laforet P, Stojkovic T, Eymard B, Costedoat-Chalumeau N, Campana-Salort E, Tournadre A, Musset L, Bader-Meunier B, Kone-Paut I, Sibilia J, Servais L, Fain O, Larroche C, Diot E, Terrier B, De Paz R, Dossier A, Menard D, Morati C, Roux M, Ferrer X, Martinet J, Besnard S, Bellance R, Cacoub P, Arnaud L, Grosbois B, Herson S, Boyer O, Benveniste O; French Myositis Network. Anti-HMGCR autoantibodies in European patients with autoimmune necrotizing myopathies: inconstant exposure to statin. Medicine (Baltimore). 2014 May;93(3):150-157. doi: 10.1097/MD.0000000000000028.

    PMID: 24797170BACKGROUND

  • Anti-HMGCR Autoantibodies in Juvenile Idiopathic Inflammatory Myopathies Identify a Rare but Clinically Important Subset of Patients. J Rheumatol. 2017 Sep;44(9):1417. doi: 10.3899/jrheum.160871.C1. No abstract available.

    PMID: 28864670BACKGROUND

  • Liang WC, Uruha A, Suzuki S, Murakami N, Takeshita E, Chen WZ, Jong YJ, Endo Y, Komaki H, Fujii T, Kawano Y, Mori-Yoshimura M, Oya Y, Xi J, Zhu W, Zhao C, Watanabe Y, Ikemoto K, Nishikawa A, Hamanaka K, Mitsuhashi S, Suzuki N, Nishino I. Pediatric necrotizing myopathy associated with anti-3-hydroxy-3-methylglutaryl-coenzyme A reductase antibodies. Rheumatology (Oxford). 2017 Feb;56(2):287-293. doi: 10.1093/rheumatology/kew386. Epub 2016 Nov 6.

    PMID: 27818386BACKGROUND

  • Tiniakou E, Pinal-Fernandez I, Lloyd TE, Albayda J, Paik J, Werner JL, Parks CA, Casciola-Rosen L, Christopher-Stine L, Mammen AL. More severe disease and slower recovery in younger patients with anti-3-hydroxy-3-methylglutaryl-coenzyme A reductase-associated autoimmune myopathy. Rheumatology (Oxford). 2017 May 1;56(5):787-794. doi: 10.1093/rheumatology/kew470.

    PMID: 28096458BACKGROUND

  • Binns EL, Moraitis E, Maillard S, Tansley S, McHugh N, Jacques TS, Wedderburn LR, Pilkington C, Yasin SA, Nistala K; UK Juvenile Dermatomyositis Research Group (UK and Ireland). Effective induction therapy for anti-SRP associated myositis in childhood: A small case series and review of the literature. Pediatr Rheumatol Online J. 2017 Oct 31;15(1):77. doi: 10.1186/s12969-017-0205-x.

    PMID: 29089059BACKGROUND

Central Study Contacts

Irène LEMELLE

CONTACT

03 83 15 45 96i.lemelle@chru-nancy.fr

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 9, 2020

First Posted

March 5, 2020

Study Start

March 1, 2020

Primary Completion

November 1, 2020

Study Completion

November 1, 2020

Last Updated

March 5, 2020

Record last verified: 2020-01

Locations

FR(1)